ET140203 T Cells in Pediatric Subjects With Hepatoblastoma, HCN-NOS, or Hepatocellular Carcinoma

NCT04634357 | Recruiting Phase 1 FDA Drug

• 1 other identifier: ETUS20AFPAR123
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

Brief Summary

Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who are AFP-positive/HLA-A2-positive and have relapsed/refractory HB, HCN-NOS, or HCC.

Conditions

Hepatoblastoma; Hepatocellular Carcinoma (HCC); Liver Neoplasms; Metastatic Liver Cancer; Liver Cancer; HEMNOS

Keywords

Relapsed/Refractory Hepatoblastoma (HB); Pediatric; Hepatocellular Neoplasm-Not Otherwise Specified (HCN-NOS); Hepatocellular Carcinoma (HCC); Liver Cancer; T-cell therapy; Metastatic Liver Cancer; Liver neoplasms; HEMNOS

Outcome Measures

Primary Outcomes (4)

• Incidence rates of adverse events (AEs) after infusion of ET140203 T cells

  Safety of ET140203 T cells as assessed by the number of adverse events (AEs) after infusion

  28 days

• Severity rates of adverse events (AEs) after infusion of ET140203 T cells

  Safety of ET140203 T cells as assessed by the severity of adverse events (AEs) after infusion.

  28 days

• Incidence rates of dose limiting toxicities (DLTs) after infusion of ET140203 T cells

  Tolerability of ET140203 T cells after infusions assessed by committee review of dose limiting toxicities (DLTs)

  28 days

• The recommended phase 2 dose (RP2D) regimen of ET140203 T cell therapy primarily based on DLT

  The RP2D will be determined by the study Dose Escalation Committee (DEC) and primarily based on DLTs.

  Up to 2 years

Secondary Outcomes (2)

• Assess the efficacy of ET140203 T cells in pediatric subjects with relapsed/refractory HB, HCN-NOS, or HCC

  Up to 2 years

• Determine the pharmacokinetics of ET140203 T cells after infusion.

  Up to 2 years

Study Arms (1)

ET140203 T Cells

EXPERIMENTAL

ET140203 Autologous T Cells

Drug: ET140203 T Cells

Interventions

ET140203 T Cells DRUG

Biological/Vaccine: ET140203 autologous T-cell product Autologous T cells transduced with lentivirus encoding an ET140203 expression construct

ET140203 T Cells

Eligibility Criteria

• Age: 1 Year - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Histologically confirmed HB, HCN-NOS, or HCC with serum AFP \>100ng/mL at the time of screening and following the most recent line of therapy.
• Disease reoccurrence after remission following initial standard-of care (SOC) treatment (i.e., relapse) or failure of response to SOC treatment (i.e., refractory).
• Age ≥ 1 year and ≤ 21 years.
• Molecular Human Leukocyte Antigen (HLA) class I allele typing that confirms subject carries at least one HLA-A2 allele.
• Life expectancy of \> 4 months per the Investigator's opinion.
• Lansky or Karnofsky Performance Scale ≥ 70.
• For enrollment to the dose-finding cohort, subjects must have at least one (1) lesion ≥ 5 mm in diameter or two (2) or more lesions ≥ 3 mm in diameter. For the dose-expansion cohort, subjects must have measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.
• Child-Pugh score of A6 or better.
• Adequate organ function.

You may not qualify if:

• Recurrent HB who are candidates for complete surgical resection (e.g., isolated pulmonary relapse amendable to pulmonary metastasectomy).
• Pre-existing illness including heart failure, uncontrolled pulmonary disease not cancer-related, or psychiatric illness/social situation that would limit compliance with study requirements.
• Active, uncontrolled systemic bacterial, fungal, or viral infection. Subjects with Human Immunodeficiency Virus (HIV), hepatitis B, or hepatitis C are eligible provided their infection is being treated and the viral load is controlled.
• Any known active malignancy (other than HB, HCN-NOS, or HCC).
• Pregnant or lactating women.
• Received the following within two (2) weeks of leukapheresis or within two (2) weeks of conditioning chemotherapy: cytotoxic chemotherapy, radiation, other anti-cancer therapies (including immunotherapeutic agents), immunosuppressive therapy, or systemic corticosteroids at doses greater than 5 mg/day of prednisone or equivalent doses of other corticosteroids. (Note: Topical and inhaled corticosteroids in standard doses and physiological replacement doses of corticosteroids for adrenal insufficiency are allowed).
• Concurrently receiving other investigational agents, biological, chemical, or radiation therapies, while participating in the study.
• Contraindication for receipt of conditioning chemotherapeutic agents including Fludarabine and Cyclophosphamide.
• Active autoimmune disease requiring systemic immunosuppressive therapy.
• Compromised circulation in the main portal vein, hepatic vein, or vena cava due to partial or complete obstruction which, in the opinion of the Investigator, would make the subject unsuitable for the study.
• History of organ transplant.
• HB, HCN-NOS, or HCC involving greater than 50% of the liver (volumetric).

Sponsors & Collaborators

• Eureka Therapeutics Inc.: lead

Study Sites (2)

UCSF Benioff Children's Hospitals

San Francisco, California, 94158, United States

RECRUITING

Dana-Farber/Boston Children's Cancer and Blood Disorders Center

Boston, Massachusetts, 02215, United States

RECRUITING

MeSH Terms

Conditions

Hepatoblastoma; Carcinoma, Hepatocellular; Liver Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms, Complex and Mixed; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Study Officials

• Pei Wang, PhD

  Eureka Therapeutics Inc.

  STUDY DIRECTOR

Central Study Contacts

Teresa Klask, MBA

CONTACT

510-722-8719; Teresa.Klask@eurekainc.com

Pei Wang, PhD

CONTACT

510-654-7045; Pei.Wang@eurekainc.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 4, 2020
• First Posted: November 18, 2020
• Study Start: July 19, 2022
• Primary Completion: January 31, 2026
• Study Completion (Estimated): January 31, 2028
• Last Updated: April 9, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)